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Label indication EMFLAZA is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. EMFLAZA is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older ( 1 ) FDA label
Also known as DFZ
Deflazacort · 4 trials · 3 indications
The area under the plasma concentration versus time curve over the final dosing interval for steady state pharmacokinetics on Day 8 of deflazacort and 21-desacetyl-DFZ, the active
Long-term safety and tolerability will be characterized by the number, frequency, and severity of adverse events from Day 1 through End of Study or Early Termination.
| Arm | Type | Description |
|---|---|---|
| Deflazacort | EXPERIMENTAL | This is an open label and single period study , dosed with 0.9mg/kg Deflazacort. |
| Hepatic Impairment | EXPERIMENTAL | Eight (8) subjects with moderate hepatic insufficiency (a score of 7 to 9, on the Child-Pugh scale) will receive one 18 mg dose of deflazacort |
| Healthy Volunteer | EXPERIMENTAL | Eight (8) healthy subjects. Subjects will be matched for age \[± 15 years\], BMI \[± 15 %\], and gender \[1:1\] to the subjects in the moderate hepatic impaired cohort; will receive 18 mg dose of deflazacort |
| Renal Impairment | EXPERIMENTAL | Eight (8) subjects with ESRD on HD will receive one 18 mg dose of deflazacort. |
| Healthy Volunteers | EXPERIMENTAL | Eight (8) healthy subjects with estimated creatinine clearance (CLcr) ≥ 90 mL/min. Subjects will be matched for age \[± 15 years\], BMI \[± 15 %\], and gender \[1:1\] to the subjects in the ESRD cohort. Subjects will receive one 18 mg dose of deflazacort. |
| Name | Type | Description |
|---|---|---|
| Deflazacort | DRUG | Oral deflazacort administered once daily at 0.9 mg/kg for eight days |
Inclusion Criteria: * In the opinion of the investigator, the subject is capable of understanding and complying with protocol requirements. * The subject or, when applicable, the subject's legally acceptable representative signs and dates a written, informed consent form and any required privacy au...
Deflazacort is studied for Duchenne Muscular Dystrophy, a genetic muscle-wasting disease, and has also been evaluated in people with hepatic impairment and renal impairment. PTC Therapeutics is developing it, and its clinical program includes Phase 1 studies in these patient groups.
Deflazacort targets NR3C1, the glucocorticoid receptor, and acts as an agonist at this receptor. This target class places it among small-molecule glucocorticoid receptor agonists, a mechanism relevant to its evaluation in Duchenne Muscular Dystrophy and in pharmacokinetic studies of hepatic and renal impairment.
PTC Therapeutics, Inc., which trades under the ticker PTCT, is the developer of Deflazacort. The company has sponsored the clinical studies evaluating the drug in Duchenne Muscular Dystrophy and in adults with hepatic or renal impairment.
Deflazacort is in Phase 1 development. It is investigational and has not been approved based on the studies described here. The program includes completed Phase 1 trials in Duchenne Muscular Dystrophy, hepatic impairment, and renal impairment, with no active trials currently listed.
Deflazacort has been studied in completed Phase 1 trials including NCT02295748, a long-term extension study in Duchenne Muscular Dystrophy; NCT02286622 in renal impairment; NCT02286609 in moderate hepatic impairment; and NCT02251600, a pharmacokinetic study in children and adolescents with Duchenne Muscular Dystrophy.